Ocular Therapeutix, Inc. has initiated the first pivotal phase 3 clinical trial assessing its axitinib intravitreal implant (OTX-TKI) for the treatment of wet age-related macular degeneration (AMD).
Let’s start with a refresher.
Ocular Therapeutix is focused on formulating, developing, and commercializing therapies for ophthalmic diseases and conditions via a proprietary bioresorbable hydrogel-based formulation technology.
Of note, DEXTENZA (dexamethasone ophthalmic insert) 0.4 mg is the biopharmaceutical company’s first FDA-approved commercial drug product.
The corticosteroid is intended for the treatment of ocular inflammation and pain post-ophthalmic surgery as well as ocular itching associated with allergic conjunctivitis.
Now OTX-TKI.
OTX-TKI is an investigational, bioresorbable hydrogel intravitreal implant with anti-angiogenic properties and designed to continuously deliver axitinib (a potent tyrosine kinase inhibitor [TKI]) for wet AMD.
It is also currently in development for the potential treatment of:
- Diabetic retinopathy (DR) (for details on the HELIOS trial, click here)
- Other vascular endothelial growth factor (VEGF)-mediated retinal diseases
How is the implant delivered?
The intravitreal implant delivers axitinib using Ocular Therapeutix’s proprietary Elutyx technology platform—a bioresorbable polymer matrix hydrogel developed to provide localized, sustained drug delivery.
OTX-TKI is administered via a 25G needle, with a target release of 9 to 12 months.
Any clinical data on it yet?
Yup! In June 2023, the company released 12-month topline data from its U.S.-based, multicenter, randomized, controlled phase 1 study (NCT04989699), in which OTX-TKI was evaluated for its safety, durability, and efficacy against aflibercept injections.
See here for the details on that promising data.
Now this pivotal trial.
The multi-center, parallel-group, superiority-designed (according to the company) trial is enrolling an estimated 300 patients with wet AMD who are treatment naïve in the study eye.
Participants will be randomized to receive either one injection of aflibercept or one implant of OTX-TKI followed by an as-needed supplemental anti-VEGF treatment based on pre-specified criteria.
Any word on who’s heading it up?
Yes, actually. Arshad M. Khanani, MD, MA, FASRS, a retina specialist, is reported to be the principal investigator.
Dr. Khanani is currently the director of Clinical Research at Sierra Eye Associates in Reno, Nevada, and has extensive experience serving as principal investigator for wet AMD and retinal disease-focused clinical trials. .
And what’s being measured?
Similar to the previous wet AMD trial, the phase 3 trial will focus on two outcomes, each measured at 36 weeks:
- Best-corrected visual acuity (BCVA)
- Central subfield thickness (CST)
Anything else of interest?
The company has requested a Special Protocol Assessment (SPA) from the FDA regarding the trial’s design as a superiority trial.
This indicates that the trial’s goal is to show that one treatment is clinically better than the other by demonstrating the “superiority” of the test agent (in this case, OTX-TKI) over placebo or the active treatment (in this case, aflibercept).
When might patient enrollment begin?
According to Ocular Therapeutix CEO Anthony Mattessich, the company plans to start enrollment before the end of 2023.
Of importance: enrollment will not begin before Ocular Therapeutix receives SPA feedback from the FDA.
Gotcha. And the significance?
OTX-TKI could potentially fill the unmet need for a retinal disease treatment (wet AMD) that provides efficacy with just a single injection and extends beyond 3 to 4 months.